Efficacy and Safety of Midostaurin in Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia

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This study will investigate if the drug midostaurin taken orally twice daily is effective and safe in treating patients with aggressive systemic mastocytosis or mast cell leukemia with or without an additional hematological neoplasm.

Midostaurin was administered at a dose of 100 mg twice daily (bid) in continuous cycles of 28 days until disease progression, intolerable toxicity or withdrawal due to any cause, whichever occurred first.

The ORR was defined as the percentage of participants who classified as confirmed responders (major response (MR) or partial response (PR). A major responder had complete resolution of at least one C-Finding and no progression in other C-Findings. A partial responder showed a measurable improvement in one or more C-Finding(s) without confirmed progression in other C-Findings. A C-Finding was a Clinical Finding, which was considered by the investigator and corroborated by the Study Steering Committee (SSC) Chairperson or designee, attributable to the mast cell disease component and not the associated hematological clonal non-mast cell lineage disease (AHNMD) component or any other cause.

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Ages Eligible for Study:

18 Years and older (Adult, Senior)

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

No

Criteria

Key Inclusion Criteria:

Confirmed diagnosis of aggressive systemic mastocytosis (ASM) or mast cell leukemia (MCL) according to WHO criteria for SM and established criteria for ASM and MCL (Valent et al 2003), presenting with at least one measurable C-Finding.

ECOG performance status of 0-3

Life expectancy > 12 weeks

ECG: QTc interval ≤ 450 ms

Meeting the following laboratory values:

AST and ALT must be ≤ 5 x Upper Limit of Normal (ULN) if this elevation is solely due to ASM/MCL, otherwise AST, ALT must be ≤ 2.5 x ULN

Serum Bilirubin must be ≤ 3 x Upper Limit of Normal (ULN) if this elevation is solely due to ASM/MCL, otherwise serum bilirubin must be

Patients with any pulmonary infiltrate including those suspected to be of infectious origin. Exception: Patients with a pleural effusion related to the disease under study as confirmed by the investigator are permitted to enter the study

Patients who have demonstrated relapse to 3 or more prior regimens of SM treatment (not including those given for supportive care)

Patients who have received any investigational agent, chemotherapy, interferon-α, or 2-chlorodeoxyadenosine (2-CdA, cladribine) within 30 days prior to first dose

Patients who have ASM with eosinophilia and known positivity for the FIP1L1-PDGFRα fusion unless they have demonstrated relapse or disease progression on prior imatinib therapy

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com